Lumacaftor: Phase III data

Vertex reported data from the identical, double-blind, international Phase III TRAFFIC and TRANSPORT trials in 1,108 CF patients ages >=12 who have 2 copies of the delta F508 CFTR mutation showing that once-daily 600 mg lumacaftor plus twice-daily 250 mg Kalydeco ivacaftor and twice-daily 400 mg lumacaftor plus twice-daily 250 mg Kalydeco each met the primary endpoint in both trials of improving the mean absolute change in percent predicted FEV1 from baseline to week 24 vs. placebo. For all endpoints, the threshold for statistical significance was a p-value of <=0.025. TRAFFIC enrolled 549 patients and TRANSPORT enrolled 559 patients.

In TRAFFIC, the difference from placebo in mean absolute change in percent predicted FEV1 was 4 percentage points in the once-daily combination arm (p<0.0001) and 2.6 percentage points in the twice-daily combination arm (p=0.0003). The difference from placebo in mean relative change in percent predicted FEV1 was 6.7% in the once-daily combination arm (p<0.0001) and 4.3% in the twice-daily combination arm (p=0.0006). In TRANSPORT, the difference from placebo in mean absolute change in percent predicted FEV1 was 2.6 percentage points in the once-daily combination arm (p=0.0004) and 3 percentage points in the twice-daily combination arm (p<0.0001). The difference from placebo in mean relative change in percent predicted FEV1 was 4.4% in the once-daily combination arm (p=0.0007) and 5.3% in the twice-daily combination arm (p<0.0001). ...